ruxolitinib for the treatment of t-cell large granular lymphocytic leukemia

Conditions: T-Cell Large Granular Lymphocyte Leukemia

Summary: this phase ii trial tests whether ruxolitinib works to shrink tumors in patients with t-cell large granular lymphocyte leukemia. ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

primary objective: i. determine the overall response rate (orr) of ruxolitinib in patients with t-cell large granular lymphocytic leukemia (t-lgll) as compared to historical controls. secondary objectives: i. rate of conversion from pr at 4 months to cr at 8 and 12 months (at full ruxolitinib dosage). ii. rate of molecular remission (t-cell receptor \[tcr\] clearance, stat3 mutation clearance) at 4, 8, 12 months. iii. incidence of grade iii/iv toxicities (at full ruxolitinib dosage). iv. quality of life using the european organization for research and treatment of cancer (eortc) quality of life questionnaire (qlq)-core 30 (c30), health assessment questionnaire-disability index (haqdi), short form (sf)-36 questionnaire at baseline, after 5 months, 1 year of treatment. exploratory objective: i. objective benefit (ob) rate at 4 months defined as a patient that had improvement in their cytopenias, transfusion dependence but not attaining a pr. ii. leukemia-free survival iii. progression-free survival outline: patients receive ruxolitinib orally (po) twice daily (bid) on days 1-28. cycles repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity. patients who achieve a response (cr or pr) may receive an additional 12 months of ruxolitinib, for a maximum of 24 months.

Eligibility criteria:
inclusion criteria: * age 18 or older and able to swallow pills * diagnosis of t-lgll defined as: cd3+cd8+ cell population \> 650/mm\^3 or cd3+cd8+cd57+ population \> 500/mm\^3 and the presence of a clonal t-cell receptor (within 1 month of diagnosis). note: patients with mds-like t-lgll may be included with pi approval even if cd3+cd8+ cell population is \ 500 cells/mm\^3 * untreated t-lgll or failed at least one line of frontline therapy; * patients must be off treatment for at least 14 days or 5 half-lives, whichever is longer * require treatment for t-lgll (one or more required) * symptomatic anemia with hemoglobin \< 10 g/dl * transfusion-dependent anemia * neutropenia with absolute neutrophil count (anc) \< 500/mm\^3 * neutropenia with anc \< 1500/mm\^3 with recurrent infections * platelet count \> 50 x 10\^9/l. platelet transfusion may be utilized to meet inclusion criteria, as long as the platelet count remains \>50,000/ul within 5 days of last transfusion. note: patients with platelets \<100 x 109/l and renal impairment are not permitted to enroll to the study. renal impairment is defined as creatinine clearance (crcl) \< 90 ml/min. * serum creatinine =\< 2 x the upper limit of normal (uln) * - estimated glomerular filtration rate (egfr) =\> 30 ml/min using the modification of diet in renal disease (mdrd) equation (multiplying egfr by each subjects body surface area \[bsa\]) * total bilirubin =\ 1.5 x uln permitted) * aspartate aminotransferase (ast) and alanine aminotransferase (alt) =\< 2.5 x uln * alkaline phosphatase (alp) =\< 2.5 x uln * eastern cooperative oncology group (ecog) performance status =\< 2 * men and women of reproductive potential must agree to follow accepted birth control methods for the duration of the study. female subject is either post-menopausal or surgically sterilized or willing to use an acceptable method of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide, or abstinence) for the duration of the study treatment until 5 half-lives have passed. male subject agrees to use an acceptable method for contraception for the duration of the study treatment until 5 half-lives have passed. * able to sign informed consent exclusion criteria: * absolute neutrophil count (anc) less than 100/mm\^3 * active infection requiring ongoing anti-microbial treatment. patients with human immunodeficiency virus (hiv), positive hepatitis b surface antigen or hepatitis c antibody will be excluded * concurrent immune-suppressive therapy (prednisone or equivalent up to 20 mg permitted to treat t-lgl symptoms, but must be weaned within one month of initiation of trial drug). patients on stable, chronic prednisone =\< 10 mg for rheumatologic/autoimmune conditions are exempted from this requirement. they may enroll on the study * active, concurrent malignancy unless deemed related to t-lgll by principal investigator (pi). early stage skin cancers, prostate cancer, permitted if under no active therapy * for females of childbearing potential: positive pregnancy test or lactating * unstable angina or myocardial infarction within the past 2 months * chronic obstructive pulmonary disease or other interstitial lung disease in active exacerbation * cirrhosis